RecruitingPhase 1

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

A Phase 1 Dose Evaluation Study of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Subjects With Refractory Autoimmune Disease

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 70 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This early study tests the safety of CTX112 at increasing doses and whether it may help adults with autoimmune diseases that have not responded to treatment. It is for people with lupus, systemic sclerosis, or certain muscle inflammation diseases, where the immune system attacks the body.

This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 18 to 69 years old.
  • Choose to sign a consent form and be willing and able to follow study visits, treatments, tests, and other rules.
  • Have blood counts and kidney, liver, heart, and lung function that meet study requirements.
  • Agree to use birth control methods allowed by the study.
  • Have lupus diagnosed by a rheumatologist, a doctor who treats autoimmune diseases, using required standards. If you have lupus kidney disease, a tissue sample must show active disease of the required type and level.
  • Or have systemic sclerosis, which causes skin and sometimes organ scarring, of a type that meets study standards. You must have active skin or lung disease.
  • Or have an allowed type of inflammatory muscle disease that meets study standards, including required levels of disease activity or skin or lung problems.

Who can't join

  • Past anti-CD19 treatment, which targets certain immune cells; gene therapy; genetically changed cell therapy; or an organ or blood-forming cell transplant.
  • Severe current or past brain or spinal cord problems; a history of seizures, stroke, dementia, or disease of the brain's balance center; or other autoimmune diseases affecting the brain or spinal cord.
  • Mixed connective tissue disease with no clear main disease, or health problems or treatments that could make the study unsafe or its results hard to judge.
  • A history of an immune system deficiency, or certain current or past bacterial, viral, or fungal infections.
  • Cancer within the past 5 years, unless it is considered unlikely to return.
  • A genetic disorder linked to bone marrow failure, or myelodysplastic syndrome, a disorder in which bone marrow makes abnormal blood cells.
  • Current or past catastrophic antiphospholipid syndrome, which causes widespread blood clots, or antiphospholipid syndrome that needs ongoing blood thinners.
  • Being pregnant or breastfeeding.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Key Inclusion Criteria: 1. Age ≥18 years and \< 70 years of age. 2. Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements. 3. Adequate hematologic, renal, liver, cardiac and pulmonary organ function. 4. Subjects must agree to use acceptable methods of contraception. 5. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures. 6. Diagnosis of systemic lupus erythematosus (SLE), systemic sclerosis (SSc) or idiopathic inflammatory myopathy (IIM). For systemic lupus erythematosus (SLE) subjects: \- Diagnosis of SLE by a board-certified rheumatologist that conforms with 2019 ACR/EULAR criteria. For lupus nephritis subjects, active, biopsy-proven proliferative lupus nephritis Class III or IV, either with or without the presence of Class V, and appropriate National Institutes of Health index activity score using the 2018 International Society of Nephrology/Renal Pathology Society criteria. For Systemic Sclerosis (SSc) subjects: \- Diagnosis of diffuse cutaneous systemic sclerosis (dcSSC) or SSc-ILD that conforms with 2013 ACR/EULAR criteria. Subjects should meet active skin or lung disease criteria. For Idiopathic Inflammatory Myopathy (IIM) subjects: \- Diagnosis with dermatomyositis (DM), polymyositis (PM) or myositis as part of rheumatologic overlap syndrome, antisynthetase (ASyS), or immune-mediated necrotizing myopathy (IMNM) that conforms with 2017 ACR/EULAR criteria for inflammatory myopathies. Subjects must meet moderate severe, skin, or lung involvement criteria. Key Exclusion Criteria: 1. Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy. 2. Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant. 3. Severe active or history of central nervous (CNS) involvement. 4. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement other than SLE, SSc or IIM. 5. Mixed connective tissue disease with no clear predominant disease. 6. Presence of study disease manifestations or other conditions that are likely to pose increase safety risks and/or confound disease assessments, or pose significant risk to those receiving CAR T cell therapy. 7. History of primary or secondary immunodeficiency. 8. Presence or history of certain bacterial, viral or fungal infection. 9. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence). 10. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome. 11. History or current diagnosis of catastrophic anti-phospholipid syndrome or anti phospholipid syndrome that requires ongoing anticoagulation. 12. Pregnant or lactating. 13. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.

Study sites (9)

CA

IA

IL

LA

MA

MO

NC

OR

  • Oregon Health Sciences University · Portland, OR 97239Richard Maziarz, MD · Principal investigatorMore trials in Portland